Clinical trial • Phase II • Dermatology|Immunology

Aldesleukin for Primary sclerosing cholangitis | Mucous membrane pemphigoid | Polymyositis/dermatomyositis | Pemphigus vulgaris/foliaceus

Phase II trial of Aldesleukin for Primary sclerosing cholangitis | Mucous membrane pemphigoid | Polymyositis/dermatomyositis | Pemphigus vulgaris/foliaceu…

Overview

Trial Therapeutic Area
Dermatology|Immunology
Trial Disease
Primary sclerosing cholangitis | Mucous membrane pemphigoid | Polymyositis/dermatomyositis | Pemphigus vulgaris/foliaceus
Trial Stage
Phase II
Drug Modality
Peptide/protein/enzyme|Small molecule
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
26-01-2024
First CTIS Authorization Date
04-04-2024

Trial design

Randomised, placebo (glucose 5%) — subcutaneous placebo administered matching the aldesleukin schedule: daily subcutaneous injections for five consecutive days (induction) followed by seven weekly subcutaneous injections until end-of-treatment at day 57 (+/-1).-controlled Phase II trial across 1 site in Germany.

Randomised
Yes
Comparator
Placebo (Glucose 5%) — subcutaneous placebo administered matching the aldesleukin schedule: daily subcutaneous injections for five consecutive days (induction) followed by seven weekly subcutaneous injections until End-of-Treatment at day 57 (+/-1).
Target Sample Size
112
Trial Duration For Participant
57

Eligibility

Recruits 112 Vulnerable population selected (isVulnerablePopulationSelected = true). Subject information and informed consent forms are listed among trial documents (e.g. L1_SIS and ICF_Main Study_GER_Redacted; L2_SIS and ICF_Accessory Biological Sampling_GER_Redacted; L3_SIS and ICF_Skin Biopsy_GER; L4_Information Leaflet_GER). No specific consent/assent handling details or age-specific consent procedures are provided in the available CTIS metadata..

Vulnerable Population
Vulnerable population selected (isVulnerablePopulationSelected = true). Subject information and informed consent forms are listed among trial documents (e.g. L1_SIS and ICF_Main Study_GER_Redacted; L2_SIS and ICF_Accessory Biological Sampling_GER_Redacted; L3_SIS and ICF_Skin Biopsy_GER; L4_Information Leaflet_GER). No specific consent/assent handling details or age-specific consent procedures are provided in the available CTIS metadata.

Inclusion criteria

  • {"criterion_text":"- Participants with pemphigus vulgaris / foliaceus (PV/PF) and having mild to moderate disease activity as defined by a PDAI ≥ 6 and ≤ 45."}
  • {"criterion_text":"- Participants with mucous membrane pemphigoid (MMP) and having mild to moderate disease activity as defined by a MMPDAI of ≥ 6 and ≤ 45."}
  • {"criterion_text":"- Participants with polymyositis (PM) or dermatomyositis (DM) and having elevated plasma concentrations of the creatine kinase (CK) of ≥ 300 U/L and ≤ 3000 U/L. Concomitant diagnoses of other rheumatic diseases (overlaps) are allowed."}
  • {"criterion_text":"- Participants with primary sclerosing cholangitis (PSC) and having elevated plasma concentrations of the alkaline phosphatase (AP) of ≥ 1.5 times above the upper limit of normal. Concomitant diagnosis of inflammatory bowel diseases is allowed."}
  • {"criterion_text":"- Age of participants: ≥ 18 years and ≤ 80 years."}

Exclusion criteria

  • {"criterion_text":"- Severe impairment of vital organ or life-threatening disease"}
  • {"criterion_text":"- Chronically active infectious diseases"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- The IL-2 induced Treg response, defined as the increase in absolute numbers of CD3+CD4+FoxP3+CD127loCD25hi Treg according to the area under the curve (AUC) obtained between day 1 and day 57,","definition_or_measurement_approach":"Defined as increase in absolute numbers of CD3+CD4+FoxP3+CD127loCD25hi Treg; measured as area under the curve (AUC) between day 1 and day 57."}
  • {"endpoint_text":"- The clinical response at day 57 assessed by absolute changes in disease-specific and z-standardized disease activity measures from day 1 to day 57. The respective disease activity measures for each of the investigated diseases are the Pemphigus Disease Area Index (PDAI), Mucous Membrane Pemphigoid Disease Area Index (MMPDAI), plasma levels of Creatine-Kinase (CK) or Alkaline Phosphatase (AP).","definition_or_measurement_approach":"Clinical response at day 57 measured by absolute changes in disease-specific and z-standardized measures from day 1 to day 57. Disease-specific measures: PDAI (pemphigus), MMPDAI (mucous membrane pemphigoid), plasma CK (polymyositis/dermatomyositis), plasma AP (PSC)."}

Secondary endpoints

  • {"endpoint_text":"- The clinical response at day 57, assessed by absolute and relative changes in disease-specific measures (PDAI, MMPDAI, CK, AP) between day 1 and day 57 compared with placebo.","definition_or_measurement_approach":"Assessed by absolute and relative changes in disease-specific measures (PDAI, MMPDAI, CK, AP) from day 1 to day 57 compared with placebo-treated participants."}

Recruitment

Planned Sample Size
112
Recruitment Window Months
36
Consent Approach
Informed consent documents are included among published trial documents (e.g. L1_SIS and ICF_Main Study_GER_Redacted; L2_SIS and ICF_Accessory Biological Sampling_GER_Redacted; L3_SIS and ICF_Skin Biopsy_GER; L4_Information Leaflet_GER). Participants are adults (≥18 years) and provide informed consent. Documents available in German (document titles indicate German language). No details on assent or multi-language consent forms are provided in the CTIS metadata.

Geography

Total Number Of Sites
1
Total Number Of Participants
112

Germany

Earliest CTIS Part Ii Submission Date
12-03-2024
Latest Decision Or Authorization Date
15-01-2025
Processing Time Days
309
Number Of Sites
1
Number Of Participants
112

Sites

Site Name
Universitaetsklinikum Schleswig-Holstein AöR
Department Name
Rheumatology and Clinical Immunology
Principal Investigator Name
Jens Humrich
Principal Investigator Email
jens.humrich@uksh.de
Contact Person Name
Jens Humrich
Contact Person Email
jens.humrich@uksh.de
Number Of Participants
112

Sponsor

Primary sponsor

Full Name
Universitaetsklinikum Schleswig-Holstein AöR
Organisation Type
Hospital/Clinic/Other health care facility
Country Of Registered Address
Germany

Third parties

  • {"country":"","full_name":"Deutsche Forschungsgemeinschaft (DFG)","duties_or_roles":"Source of monetary support","organisation_type":""}

Investigational products

Investigational Product Name
PROLEUKIN® S 18 x 106 IE Pulver zur Herstellung einer Injektionslösung oder Infusionslösung
Active Substance
Aldesleukin
Modality
Peptide/protein/enzyme
Routes Of Administration
Subcutaneous
Route
Subcutaneous
Authorisation Status
Marketing authorisation exists (marketingAuthNumber: 17152.00.00)
Orphan Designation
Yes
Starting Dose
1.5 million IU (1,500,000 IU) subcutaneously (induction schedule)
Dose Levels
1.5 million IU (single dose level used in study)
Frequency
Daily for five consecutive days (induction), followed by seven weekly injections (maintenance) until day 57 (+/-1)
Maximum Dose
Max daily dose 1,500,000 IU; max total dose amount 18,000,000 IU
Investigational Product Name
GLUCOSE (placebo)
Active Substance
Glucose
Modality
Small molecule
Routes Of Administration
Subcutaneous
Route
Subcutaneous
Authorisation Status
No marketing authorisation (marketingAuthNumber: -)
Starting Dose
Placebo administered matching aldesleukin schedule; maximum daily volume up to 10 ml as per product data
Frequency
Matched to aldesleukin: daily for five days then weekly for seven weeks until day 57
Maximum Dose
Max daily volume 10 ml (per product entry)

Related trials

Other published trials that may interest you.